Tongue and Hypoglossal Motoneuron Gene Therapy in a Rat Model of Pompe Disease

被引：1
|
作者：
Singer, Michele
Rana, Sabhya
Pope, Megan
Patterson, LiAna
Corti, Manuela
Byrne, Barry
Fuller, David
机构：
来源：
FASEB JOURNAL | 2021年 / 35卷
关键词：
D O I：
10.1096/fasebj.2021.35.S1.02822
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
引用
收藏
页数：2
相关论文
共 50 条
  • [11] Lentiviral gene therapy for Pompe disease
    Stok, M.
    de Boer, H.
    de Visser, T. P.
    Reuser, A. J.
    Jacobs, E.
    Jahr, H.
    Duncker, D.
    van Til, N. P.
    Wagemaker, G.
    HUMAN GENE THERAPY, 2011, 22 (10) : A40 - A41
  • [12] Gene Therapy Developments for Pompe Disease
    Unnisa, Zeenath
    Yoon, John K.
    Schindler, Jeffrey W.
    Mason, Chris
    van Til, Niek P.
    BIOMEDICINES, 2022, 10 (02)
  • [13] Tongue weakness in Pompe disease
    Dubrovsky, A.
    Corderi, J. C.
    NEUROMUSCULAR DISORDERS, 2008, 18 (9-10) : 803 - 803
  • [14] MODULATION OF NEONATAL RAT HYPOGLOSSAL MOTONEURON EXCITABILITY BY SEROTONIN
    BERGER, AJ
    BAYLISS, DA
    VIANA, F
    NEUROSCIENCE LETTERS, 1992, 143 (1-2) : 164 - 168
  • [15] Developmental changes in hypoglossal motoneuron morphology in neonatal rat
    Williams, Paul Allen
    Bellinger, Denise
    Wilson, Christopher G.
    FASEB JOURNAL, 2018, 32 (01):
  • [16] Treatment of Infantile-Onset Pompe Disease in a Rat Model with Muscle-Directed AAV Gene Therapy
    Garcia, Miquel
    Munoz, Sergio
    Bertolin, Joan
    Jimenez, Veronica
    Jaen, Maria Luisa
    Pujol, Anna
    Vila, Laia
    Sacristan, Victor
    Barbon, Elena
    Ronzitti, Giuseppe
    El Andari, Jihad
    Tulalamba, Warut
    Pham, Quang Hong
    Ruberte, Jesus
    Vandendriessche, Thierry C.
    Chuah, Marinee K. L.
    Grimm, Dirk
    Mingozzi, Federico
    Bosch, Fatima
    MOLECULAR THERAPY, 2023, 31 (04) : 785 - 785
  • [17] Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
    Munoz, Sergio
    Bertolin, Joan
    Jimenez, Veronica
    Jaen, Maria Luisa
    Garcia, Miquel
    Pujol, Anna
    Vila, Laia
    Sacristan, Victor
    Barbon, Elena
    Ronzitti, Giuseppe
    El Andari, Jihad
    Tulalamba, Warut
    Pham, Quang Hong
    Ruberte, Jesus
    VandenDriessche, Thierry
    Chuah, Marinee K.
    Grimm, Dirk
    Mingozzi, Federico
    Bosch, Fatima
    MOLECULAR METABOLISM, 2024, 81
  • [18] Progress and challenges of gene therapy for Pompe disease
    Ronzitti, Giuseppe
    Collaud, Fanny
    Laforet, Pascal
    Mingozzi, Federico
    ANNALS OF TRANSLATIONAL MEDICINE, 2019, 7 (13)
  • [19] Gene-cell therapy for Pompe disease
    Gonzalez-Correa, J. E.
    Aguilar-Gonzalez, A.
    Munoz, P.
    Martin, F.
    HUMAN GENE THERAPY, 2019, 30 (11) : A154 - A154
  • [20] Gene Therapy for Pompe Disease: The Time Is Now
    Colella, Pasqualina
    Mingozzi, Federico
    HUMAN GENE THERAPY, 2019, 30 (10) : 1245 - 1262
12345